1. Kruger K, Grabowski PJ, Zaug AJ, Sands J, Gottschling DE, Cech TR. Self-splicing RNA: autoexcision and autocyclization of the ribosomal RNA intervening sequence of Tetrahymena. Cell 1982;31(1):147-57.

2. Cech and Gestalang, The RNA World: CSHL Press, 1999.

3. Breaker RR. In Vitro Selection of Catalytic Polynucleotides. Chem Rev 1997;97(2):371-390.

4. Puerta-Fernandez E, Romero-Lopez C, Barroso-delJesus A, Berzal-Herranz A. Ribozymes: recent advances in the development of RNA tools. FEMS Microbiol Rev 2003;27(1):75-97.

5. Nissen P, Hansen J, Ban N, Moore PB, Steitz TA. The structural basis of ribosome activity in peptide bond synthesis. Science 2000;289(5481):920-30.

6. Haseloff J, Gerlach WL. Simple RNA enzymes with new and highly specific endoribonuclease activities. Nature 1988;334(6183):585-91.

7. Branch AD, Robertson HD. Efficient trans cleavage and a common structural motif for the ribozymes of the human hepatitis delta agent. Proc Natl Acad Sci U S A 1991;88(22):10163-7.

8. Pestka S, Daugherty BL, Jung V, Hotta K, Pestka RK. Anti-mRNA: specific inhibition of translation of single mRNA molecules. Proc Natl Acad Sci U S A 1984;81(23):7525-8.

9. Izant JG, Weintraub H. Constitutive and conditional suppression of exogenous and endogenous genes by anti-sense RNA. Science 1985;229(4711):345-52.

10. van der Krol AR, Mol JN, Stuitje AR. Modulation of eukaryotic gene expression by complementary RNA or DNA sequences. Biotechniques 1988;6(10):958-76.

11. Phylactou LA, Tsipouras P, Kilpatrick MW. Hammerhead ribozymes targeted to the FBN1 mRNA can discriminate a single base mismatch between ribozyme and target. Biochem Biophys Res Commun 1998;249(3):804-10.

12. Kore AR, Vaish NK, Kutzke U, Eckstein F. Sequence specificity of the hammerhead ribozyme revisited; the NHH rule. Nucleic Acids Res 1998;26(18):4116-20.

13. Beigelman L, McSwiggen JA, Draper KG, Gonzalez C, Jensen K, Karpeisky AM, et al. Chemical modification of hammerhead ribozymes. Catalytic activity and nuclease resistance. J Biol Chem 1995;270(43):25702-8.

14. Weng DE, Usman N. Angiozyme: a novel angiogenesis inhibitor. Curr Oncol Rep 2001;3(2):141-6.

15. Sullenger BA, Cech TR. Tethering ribozymes to a retroviral packaging signal for destruction of viral RNA. Science 1993;262(5139):1566-9.

16. Lee NS, Bertrand E, Rossi J. mRNA localization signals can enhance the intracellular effectiveness of hammerhead ribozymes. Rna 1999;5(9):1200-9.

17. Chirmule N, Propert K, Magosin S, Qian Y, Qian R, Wilson J. Immune responses to adenovirus and adeno-associated virus in humans. Gene Ther 1999;6(9):1574-83.

18. Niidome T, Huang L. Gene therapy progress and prospects: nonviral vectors. Gene Ther 2002;9(24):1647-52.

19. Zhang YA, Nemunaitis J, Scanlon KJ, Tong AW. Anti-tumorigenic effect of a K-ras ribozyme against human lung cancer cell line heterotransplants in nude mice. Gene Ther 2000;7(23):2041-50.

20. Mendoza-Maldonado R, Zentilin L, Fanin R, Giacca M. Purging of chronic myelogenous leukemia cells by retrovirally expressed anti-bcr-abl ribozymes with specific cellular compartmentalization. Cancer Gene Ther 2002;9(1):71-86.

21. Aigner A, Renneberg H, Bojunga J, Apel J, Nelson PS, Czubayko F. Ribozyme-targeting of a secreted FGF-binding protein (FGF-BP) inhibits proliferation of prostate cancer cells in vitro and in vivo. Oncogene 2002;21(37):5733-42.

22. Kobayashi H, Takemura Y, Miyachi H. Novel approaches to reversing anti-cancer drug resistance using gene-specific therapeutics. Hum Cell 2001;14(3):172-84.

23. Wang L, Witherington C, King A, Gerlach WL, Carr A, Penny R, et al. Preclinical characterization of an anti-tat ribozyme for therapeutic application. Hum Gene Ther 1998;9(9):1283-91.

24. Bauer G, Valdez P, Kearns K, Bahner I, Wen SF, Zaia JA, et al. Inhibition of human immunodeficiency virus-1 (HIV-1) replication after transduction of granulocyte colony-stimulating factor-mobilized CD34+ cells from HIV-1-infected donors using retroviral vectors containing anti-HIV-1 genes. Blood 1997;89(7):2259-67.

25. Wong-Staal F, Poeschla EM, Looney DJ. A controlled, Phase 1 clinical trial to evaluate the safety and effects in HIV-1 infected humans of autologous lymphocytes transduced with a ribozyme that cleaves HIV-1 RNA. Hum Gene Ther 1998;9(16):2407-25.

26. Macejak DG, Jensen KL, Pavco PA, Phipps KM, Heinz BA, Colacino JM, et al. Enhanced antiviral effect in cell culture of type 1 interferon and ribozymes targeting HCV RNA. J Viral Hepat 2001;8(6):400-5.

27. Lee PA, Blatt LM, Blanchard KS, Bouhana KS, Pavco PA, Bellon L, et al. Pharmacokinetics and tissue distribution of a ribozyme directed against hepatitis C virus RNA following subcutaneous or intravenous administration in mice. Hepatology 2000;32(3):640-6.

28. Morrissey DV, Lee PA, Johnson DA, Overly SL, McSwiggen JA, Beigelman L, et al. Characterization of nuclease-resistant ribozymes directed against hepatitis B virus RNA. J Viral Hepat 2002;9(6):411-8.

29. Dawson PA, Marini JC. Hammerhead ribozymes selectively suppress mutant type I collagen mRNA in osteogenesis imperfecta fibroblasts. Nucleic Acids Res 2000;28(20):4013-20.

30. Toudjarska I, Kilpatrick MW, Niu J, Wenstrup RJ, Tsipouras P. Delivery of a hammerhead ribozyme specifically downregulates mutant type I collagen mRNA in a murine model of osteogenesis imperfecta. Antisense Nucleic Acid Drug Dev 2001;11(5):341-6.

31. Millington-Ward S, O'Neill B, Kiang AS, Humphries P, Kenna PF, Farrar GJ. A mutation-independent therapeutic strategem for osteogenesis imperfecta. Antisense Nucleic Acid Drug Dev 1999;9(6):537-42.

32. Lewin AS, Drenser KA, Hauswirth WW, Nishikawa S, Yasumura D, Flannery JG, et al. Ribozyme rescue of photoreceptor cells in a transgenic rat model of autosomal dominant retinitis pigmentosa. Nat Med 1998;4(8):967-71.

33. Suzuki Y, Funato T, Munakata Y, Sato K, Hirabayashi Y, Ishii T, et al. Chemically modified ribozyme to V gene inhibits anti-DNA production and the formation of immune deposits caused by lupus lymphocytes. J Immunol 2000;165(10):5900-5.

34. Takahashi M, Funato T, Suzuki Y, Fujii H, Ishii KK, Kaku M, et al. Chemically modified ribozyme targeting TNF-alpha mRNA regulates TNF-alpha and IL-6 synthesis in synovial fibroblasts of patients with rheumatoid arthritis. J Clin Immunol 2002;22(4):228-36.

35. Enjoji M, Wang F, Nakamuta M, Chan L, Teng BB. Hammerhead ribozyme as a therapeutic agent for hyperlipidemia: production of truncated apolipoprotein B and hypolipidemic effects in a dyslipidemia murine model. Hum Gene Ther 2000;11(17):2415-30.